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ClinicalTrials.gov public records Last synced Sep 27, 2026, 9:59 AM EDT

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Showing 1–24 of 512 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Pediatric: Genetic Syndrome Clear all
Completed No phase listed Observational

Genetic Analysis of Oculocerebrorenal Syndrome of Lowe

NCT00359515
View directory record Official record
Conditions
Lowe Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 27, 2026, 9:59 AM EDT
Recruiting Not applicable Interventional

Gastrointestinal Response of Pediatric Cystic Fibrosis Patients on Mediterranean Diet

NCT07223255
View directory record Official record
Conditions
Cystic Fibrosis (CF), Cystic Fibrosis Gastrointestinal Disease
Interventions
Mediterranean diet
Other
Lead sponsor
Dartmouth-Hitchcock Medical Center
Other
Eligibility
3 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
2
States / cities
Lebanon, New Hampshire • Manchester, New Hampshire
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Phase 2 Interventional Results available

Aztreonam Lysine for Pseudomonas Infection Eradication Study

NCT01375049
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Aztreonam for Inhalation Solution (AZLI)
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
3 Months to 17 Years
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
23
States / cities
Phoenix, Arizona • Jacksonville, Florida • Orlando, Florida + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2014 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Phase 4 Interventional Results available

Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency

NCT01465958
View directory record Official record
Conditions
Primary Immunodeficiency
Interventions
GAMUNEX-C
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
2 Years to 16 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
4
States / cities
Los Angeles, California • Centennial, Colorado • Hollywood, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2015 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Phase 2 Interventional

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

NCT02715115
View directory record Official record
Conditions
Rett Syndrome
Interventions
NNZ-2566, Placebo
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
5 Years to 15 Years · Female only
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
12
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2020 · Synced Sep 27, 2026, 9:59 AM EDT
Active, not recruiting Phase 4 Interventional

A Study of Long-term Effect of Dupilumab on Skin Barrier Function in Pediatric Participants With Atopic Dermatitis

NCT05983068
View directory record Official record
Conditions
Dermatitis Atopic
Interventions
dupilumab
Drug
Lead sponsor
Sanofi
Industry
Eligibility
6 Years to 14 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
3
States / cities
Denver, Colorado • St Louis, Missouri • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Sep 27, 2026, 9:59 AM EDT
Withdrawn Phase 1 Interventional

A Phase I Study of Pazopanib as a Single Agent for Children With Refractory Solid Tumors

NCT01130623
View directory record Official record
Conditions
Sarcoma, Neuroblastoma, Wilms Tumor, Osteosarcoma, Brain Tumor
Interventions
Pazopanib (GW786034)
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 25 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 27, 2026, 9:59 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options

NCT02650401
View directory record Official record
Conditions
Solid Tumors, CNS Tumors
Interventions
Entrectinib
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
0 Years to 18 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2027
U.S. locations
15
States / cities
La Jolla, California • San Francisco, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Phase 1Phase 2 Interventional

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

NCT04259281
View directory record Official record
Conditions
Angelman Syndrome
Interventions
GTX-102
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
4 Years to 17 Years
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
6
States / cities
Los Angeles, California • San Diego, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Phase 3 Interventional Results available

Long Term Extension Study of Tapinarof Cream, 1% for Subjects With Atopic Dermatitis

NCT05142774
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Tapinarof cream, 1%
Drug
Lead sponsor
Organon and Co
Industry
Eligibility
2 Years and older
Enrollment
728 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
102
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 88 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2025 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Not applicable Interventional

Brief Cognitive Behavioral Therapy to Treat Itch Rumination "Itch CBT" in Eczema

NCT04486742
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Itch CBT
Behavioral
Lead sponsor
Northwestern University
Other
Eligibility
8 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Nov 8, 2023 · Synced Sep 27, 2026, 9:59 AM EDT
Active, not recruiting Phase 1 Interventional

Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas

NCT03478462
View directory record Official record
Conditions
Pediatric Solid Tumor, Pediatric Lymphoma, Pediatric Brain Tumor, DIPG, Neuroblastoma, Ewing Sarcoma, Rhabdomyosarcoma, Osteosarcoma
Interventions
CLR 131
Drug
Lead sponsor
Cellectar Biosciences, Inc.
Industry
Eligibility
2 Years to 25 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
6
States / cities
Palo Alto, California • New York, New York • Chapel Hill, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Sep 27, 2026, 9:59 AM EDT
Recruiting Not applicable Interventional

Integrative Training Program for Pediatric Sickle Cell Pain

NCT06691867
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
I-STRONG, Enhanced Usual Care (EUC)
Behavioral · Other
Lead sponsor
Emory University
Other
Eligibility
12 Years to 18 Years
Enrollment
155 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
4
States / cities
Hartford, Connecticut • Atlanta, Georgia • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 13, 2025 · Synced Sep 27, 2026, 9:59 AM EDT
Recruiting Phase 3 Interventional

BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.

NCT07605429
View directory record Official record
Conditions
Angelman Syndrome
Interventions
rugonersen, Sham procedure
Drug · Procedure
Lead sponsor
OHB Pediatrics Ltd.
Industry
Eligibility
1 Year to 50 Years
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
3
States / cities
San Diego, California • Chicago, Illinois • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Sep 27, 2026, 9:59 AM EDT
Recruiting Phase 2 Interventional

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old

NCT07536269
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Crinecerfont
Drug
Lead sponsor
Neurocrine Switzerland GmbH
Industry
Eligibility
3 Months to 47 Months
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
11
States / cities
San Francisco, California • Aurora, Colorado • Hartford, Connecticut + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 27, 2026, 9:59 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Investigate Safety and Efficacy of Tapinarof Cream, 1% in Participants Ages 3 Months to < 24 Months With Atopic Dermatitis

NCT07265479
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Tapinarof cream, 1%, Vehicle Cream
Drug
Lead sponsor
Organon and Co
Industry
Eligibility
3 Months to 23 Months
Enrollment
187 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
36
States / cities
Birmingham, Alabama • Bryant, Arkansas • Los Angeles, California + 32 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Not applicable Interventional

Feasibility and Efficacy of a Home-based, Computerized Cognitive Training Program in Pediatric Sickle Cell Disease

NCT02857023
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Cogmed RM
Behavioral
Lead sponsor
Steven J. Hardy
Other
Eligibility
7 Years to 16 Years
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Nov 22, 2018 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Phase 3 Interventional Results available

Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

NCT00006400
View directory record Official record
Conditions
Hematologic Diseases, Anemia, Sickle Cell
Interventions
Hydroxyurea, Placebo
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
9 Months to 18 Months
Enrollment
193 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
14
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2020 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Phase 2 Interventional Results available

Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients

NCT03474965
View directory record Official record
Conditions
Sickle Cell Disease (SCD)
Interventions
Crizanlizumab
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
12
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Sep 27, 2026, 9:59 AM EDT
Not listed Phase 1Phase 2 Interventional

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

NCT02061800
View directory record Official record
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 3, 2024 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Phase 2 Interventional

Liposomal Vincristine for Pediatric and Adolescent Patients With Relapsed Malignancies

NCT00038207
View directory record Official record
Conditions
Soft Tissue Sarcoma, Lymphoma, Leukemia, Wilms' Tumor, Osteosarcoma
Interventions
Liposomal Vincristine
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 30, 2018 · Synced Sep 27, 2026, 9:59 AM EDT
Terminated Phase 1 Interventional

A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74

NCT06597656
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, Plasmapheresis
Genetic · Procedure
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced Sep 27, 2026, 9:59 AM EDT
Completed Phase 2 Interventional Results available

Arginine Therapy for Sickle Cell Disease Pain

NCT02536170
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Pain Episode
Interventions
L-arginine, L-arginine Loading Dose, Placebo
Drug · Other
Lead sponsor
Emory University
Other
Eligibility
3 Years to 21 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
3
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 5, 2023 · Synced Sep 27, 2026, 9:59 AM EDT
Not listed Not applicable Interventional

FOCUS for Pediatric Sickle Cell Disease and Cancer

NCT04075877
View directory record Official record
Conditions
Pediatric Cancer, Sickle Cell Disease, Quality of Life, Depressive Symptoms, Coping Skills
Interventions
FOCUS
Behavioral
Lead sponsor
Georgia State University
Other
Eligibility
12 Years to 18 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Nov 12, 2020 · Synced Sep 27, 2026, 9:59 AM EDT